Melanoma is the deadliest form of skin cancer – claiming the lives of almost 3,000 American women every year. It’s also the fastest-growing cancer in the world, according to the National Cancer Institute. But advances in melanoma research are improving survival rates. Lifescript spoke to top doctors in the field to find out what the latest melanoma treatments mean to patients...

For years, skin cancer thwarted doctors and robbed patients. Advanced cases of the disease are especially challenging to treat, because if the cancer has spread to organs, it’s usually incurable.

But now both scientists and patients are cheering the approval of several new weapons in the fight against melanoma that can add months, or even years, to patients’ lives. This month, the Food and Drug Administration (FDA) green-lighted immunity-boosting cancer drug ipilimumab and at least two other drugs are in the final stretches of clinical trials.

“In the last 10-20 years, we’ve made amazing scientific discoveries in melanoma, and a lot of research and development is now paying off,” says Steven J. O’Day, M.D., chief of research and director of the melanoma program at The Angeles Clinic and Research Institute in Los Angeles. He was on the team that researched ipilimumab.

“Now, with a disease like metastasized melanoma – with no treatment options – we’ve suddenly broken through with two major areas [immunity and gene makeup] and many more to come,” O’Day adds.

To get the latest on this life-saving melanoma research, Lifescript spoke with O’Day and other experts on the front lines of the disease about their clinical trials and how patients can benefit from them.

How does ipilimumab (brand name: Yervoy) work against melanoma?Most therapies target the cancer and eradicate it directly. But ipilimumab targets the patient’s own T cells [immune cells] to make them stronger. Then the T cells go and kill the melanoma indirectly.

This is a major shift to an immunology [standpoint] from the previous [melanoma] treatment.

Traditionally, chemotherapy works only for months – rarely for years. Ipilimumab takes more time to work, but when it [does], it most frequently works for years and cures some patients.

It seems to [offer] long-term results [while] other treatments may work only for short periods.

Do patients have to take ipilimumab all their lives?No, that’s the beauty of this drug – just four doses over three months. Patients don’t need further treatment and they’ve been doing well for years.

[They] get the drug and then resume their normal lives; the ones benefiting – 30% – rarely need to be re-treated.

How do you know who’ll benefit from this melanoma treatment?We haven’t figured out which factor predicts for the 30% [who] seem to have long-term benefits.

The real key will be to look for immune system markers [and] be able to tell the patient, “You’re very likely to respond, so go forward with this [melanoma] treatment.” Or, “this is really not the right treatment for you. Let’s try something else.”

How many have long-term benefits? Of [the 30% who] respond, 80% seem to have long-term responses. Only about 20% of them relapsed down the road.

If they relapse later, we can treat with the same drug.

So it’s not like chemotherapy, where you have to stop administering melanoma treatment at a certain point?That’s right. You can treat for a short period and if patients benefit, great. If they benefit and relapse, you can re-treat them with the same drug, and [it will help] many of those patients a second time.

Why is boosting the immune system so important?Because the immune system is trying to control cancer and keep it from spreading. Mostly, it loses that battle to the tumor.

But if we can resurrect the [immune] system with these drugs and strengthen it, then it can win the fight.

And it won’t be restricted to melanoma or kidney cancer. We’re hoping it has applications to other hard-to-treat tumors.

Expert #2: Peter Boasberg, M.D.,physician in the melanoma program at The Angeles Clinic and Research Institute in Los Angeles

You’re doing melanoma research on two drugs – Abraxane and Avastin (funded by Celgene and Roche, Inc., respectively). What’s Abraxane?It’s really a combination of a very common cancer drug that’s used extensively in lung, ovarian and breast cancer. It’s called paclitaxel and it [binds] to a protein called albumin, which is the major protein in our [blood].

How does this melanoma treatment work?It’s transported through the [blood] into tumor cells. The albumin is digested, and paclitaxel is released right at the tumor.

The albumin is really a carrying agent and acts like a Trojan horse, because the body doesn’t see the chemotherapy – all it sees is the regular albumin.

How does Avastin work?Avastin is an antibody that binds to vascular endothelial growth factor (VEGF). Tumor cells make VEGF to stimulate blood vessels, [which] they need to grow and metastasize. If you can choke off their blood supply, the tumors languish or die.

We use Abraxane and Avastin together; they really synergized with each other.

How successful are they?[We] have a large number of patients alive for two years, which is unheard of in chemotherapy for melanoma.

What do you see in the future of melanoma treatment?With advances in molecular biology and genomics [the study of genomes], they’ll find different mutations to be targeted and inhibited. It’s just the beginning of therapy for this disease.

What’s the focus of your melanoma research?Drugs that interfere with the genetics of melanoma. The drug we’re using, vemurafenib, has shown overall improved survival. [Research is funded by drug company Roche.]

How does this experimental drug work?It blocks the production of an activated gene called BRAF, [which is in] 50% of melanoma tumors [and drives them]. It’s better than standard chemotherapy.

What’s the success rate of this melanoma treatment? There was a 63% decreased risk of death in vemurafenib patients.

How many patients reduced the risk of their cancer progressing compared to standard chemotherapy?[It] was decreased by 74%.

How many had their tumors shrink?This is interim data, but so far it was 48.4%.

What are vemurafenib’s side effects?They’re mild. The main side effects were joint pain, rash, fatigue, photosensitivity and inflammation in the liver. We also saw non-melanoma skin tumors in the vemurafenib group.

We’re hearing more about the importance of genetic testing of tumors in determining melanoma treatment. Will this be common?In the next couple of years, when pathologists look at a tumor and say it’s a melanoma, they’ll also be doing genetic tests as a routine.

A panel of genes automatically gets tested for lung and colon cancer, and very soon we’ll have that for melanoma.

There [will] be different sets of genes for different tumors. The genes that are relevant for lung cancer probably aren’t going to be the same for melanoma.

Hopefully, gene testing will determine the right treatment for each type of tumor.

It sounds like melanoma treatment is making extraordinary progress right now.The field is moving very quickly. [That’s why] patients really ought to be coming to centers of excellence for melanoma treatment, because that’s where the action is. We can also help direct patients to appropriate clinical trials.

Expert #4:Omid Hamid, M.D., associate director of the melanoma research program at The Angeles Clinic and Research Institute in Los Angeles

Which kinds of patients are you seeking for clinical trials: those who have had melanoma treatment or those who haven’t?Part of my role is to ensure that the mix of clinical trials covers everyone. That means we look for patients who have received prior therapy and those who have not – and who haven’t experienced changes in their tumors for some time – or patients with aggressive or progressive [cancer].

What would you say to people reluctant to participate in a clinic trial?We’re at your service. The physician who offers you a clinical trial is giving you a consultation. You are the main decision maker.

Any patient who is on a clinical trial can come off at any time.

Many clinical trials for melanoma treatment are not usually free. How expensive is it?There are some minimal costs for being in a clinical trial. [Most] drug companies cover a significant amount of the cost. Being able to have access to the drug and [lifesaving therapies] before anyone else is important.

Test Your Skin Cancer IQWhen it comes to your sun safety, is your skin cancer knowledge current or is ignorance bliss? Consider this: Skin cancer is diagnosed more often than all other cancers combined. Have we caught your attention now? Find out just how much you know when it comes to skin cancer and prevention.

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